Clinical Data Required for the Approval of Pharmaceuticals for Pediatrics in Japan

Abstract Background In Japan, the number of pharmaceuticals with pediatric indications is small; some of them are approved only in Europe or the USA but not in Japan. Analyses of review reports by Japanese health authorities may clarify the type of data that facilitate the approval of pediatric drugs in Japan. Methods We identified products approved for marketing authorization in Japan, extracted relevant product- and review-related information, and summarized the characteristics of pediatric drugs clinical data and drug approval procedures. Results Among 625 products (approved 04/2019–02/2024), 171 with pediatric indications were analyzed. The approval review considered orphan drug designation for 56 products, public knowledge-based application for 16 products, mandatory post-marketing surveillance for 42 products, and investigator-initiated studies for 11 products.